Experimental Gene Therapy Found To Slow Huntington's Disease Progression (bbc.com) 13
Doctors report the first successful treatment for Huntington's disease using a new type of gene therapy given during 12 to 18 hours of delicate brain surgery. The BBC reports: An emotional research team became tearful as they described how data shows the disease was slowed by 75% in patients. It means the decline you would normally expect in one year would take four years after treatment, giving patients decades of "good quality life", Prof Sarah Tabrizi told BBC News. The first symptoms of Huntington's disease tend to appear in your 30s or 40s and is normally fatal within two decades -- opening the possibility that earlier treatment could prevent symptoms from ever emerging. None of the patients who have been treated are being identified, but one was medically retired and has returned to work. Others in the trial are still walking despite being expected to need a wheelchair. Treatment is likely to be very expensive. However, this is a moment of real hope in a disease that hits people in their prime and devastates families. [...]
It starts with a safe virus that has been altered to contain a specially designed sequence of DNA. This is infused deep into the brain using real-time MRI scanning to guide a microcatheter to two brain regions - the caudate nucleus and the putamen. This takes 12 to 18 hours of neurosurgery. The virus then acts like a microscopic postman -- delivering the new piece of DNA inside brain cells, where it becomes active. This turns the neurons into a factory for making the therapy to avert their own death. The cells produce a small fragment of genetic material (called microRNA) that is designed to intercept and disable the instructions (called messenger RNA) being sent from the cells' DNA for building mutant huntingtin. This results in lower levels of mutant huntingtin in the brain. [...]
The data showed that three years after surgery there was an average 75% slowing of the disease based on a measure which combines cognition, motor function and the ability to manage in daily life. The data also shows the treatment is saving brain cells. Levels of neurofilaments in spinal fluid -- a clear sign of brain cells dying -- should have increased by a third if the disease continued to progress, but was actually lower than at the start of the trial.
It starts with a safe virus that has been altered to contain a specially designed sequence of DNA. This is infused deep into the brain using real-time MRI scanning to guide a microcatheter to two brain regions - the caudate nucleus and the putamen. This takes 12 to 18 hours of neurosurgery. The virus then acts like a microscopic postman -- delivering the new piece of DNA inside brain cells, where it becomes active. This turns the neurons into a factory for making the therapy to avert their own death. The cells produce a small fragment of genetic material (called microRNA) that is designed to intercept and disable the instructions (called messenger RNA) being sent from the cells' DNA for building mutant huntingtin. This results in lower levels of mutant huntingtin in the brain. [...]
The data showed that three years after surgery there was an average 75% slowing of the disease based on a measure which combines cognition, motor function and the ability to manage in daily life. The data also shows the treatment is saving brain cells. Levels of neurofilaments in spinal fluid -- a clear sign of brain cells dying -- should have increased by a third if the disease continued to progress, but was actually lower than at the start of the trial.
It's not a virus (Score:3)
It's the capsid of a virus, not an actual virus being used. Good way to trigger the anti-science MAGA crowd though.
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Fuck 'em. I'm fine with them opting out of medical science.
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boo fucking hoo
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The right constantly dehumanizes people and equates empathy for people outside their tribe with evil.
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This is fantastic! (Score:2)
I lost my mother, almost 5 years ago to the day, to this awful disease. This is great news! it seems like in the past year or two, there have been some great strides to get working treatments for people afflicted with Huntingtons. Stories like this really give me hope that one day, HD will be treatable at earlier ages and people won't have to go through what my mom went through and what my sister and I had to see happen to our mom.
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I lost my mother, almost 5 years ago to the day, to this awful disease. This is great news! it seems like in the past year or two, there have been some great strides to get working treatments for people afflicted with Huntingtons. Stories like this really give me hope that one day, HD will be treatable at earlier ages and people won't have to go through what my mom went through and what my sister and I had to see happen to our mom.
My condolences. A relative of mine was recently diagnosed with CFS1R adult onset leukoencephalopathy. Different mechanism of action but also progressive, uncurable and hereditary. Knowing the autosomal dominant transmission vector of Huntingtons, I hope you and your sister are unaffected. Rarer diseases by nature have less research funding, so it is indeed great to see any progress that is made.
What is slashdot doing to the article links? (Score:5, Interesting)
When I inspect the link in the article it shows an anchor with no url included and a Jquery event attached to it. What evil is slashdot up to? Is it advertising related?
Have a link anyway - https://www.bbc.com/news/artic... [bbc.com]